DISCLAIMER
The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.compose your message
message sent
email sent successfully
Trusted Resources: News & Events
Latest announcements and gatherings
Gene therapies could transform the treatment of sickle cell disease
Manny Johnson keeps catching himself rubbing a spot on the upper right side of his chest. Starting when he was a teenager, the 21-year-old from Boston depended on a port implanted there to ease his monthly blood transfusions for sickle cell disease, a genetic disorder that caused a stroke when he was three.
But in November, six months after Johnson became the first patient to receive an experimental therapy aimed at curing his disease, the port that had become part of him — requiring special approval to play sports, used when he was excused from school for a day or two every month for treatments — was removed. Johnson hasn’t needed a transfusion or had any symptoms since May.
“It’s like, wow. I’m fascinated by it,” said Johnson, who added that he almost didn’t know what to make of it when his medical team told him they wouldn’t need to see him for three whole months. “I’m so used to being next to them. It’s like: freedom.”
Related Content
-
news & eventsSickle cell disease cure in sights of UAB Stem Cell InstituteTim Townes has a dream. He has a dream t...
-
news & eventsProdigy’s death shines light on slow progress against sickle cell diseaseThe death of the rap artist Prodigy (Alb...
-
news & eventsGene-Editing Treatment Shows Promise for Sickle Cell DiseaseScientists are seeing promising early re...
-
news & eventsGene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
-
news & eventsSolution to 50-year-old mystery could lead to gene therapy for common blood disordersIn a landmark study that could lead to n...
-
news & eventsFDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe SCD and Transfusion-Dep...BOSTON & ZUG, Switzerland--(BUSINESS...
-
news & eventsBioverativ and Sangamo announce FDA acceptance of IND application for gene-edited cell therapy BIVV003 to treat sick...Bioverativ Inc., a Sanofi company dedi...
send a message
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.